June 2025:  The SMC contacted patient groups to inform us that nusinersen will shortly be assessed by the SMC for use in NHS Scotland. They have invited us to provide a Patient Group Submission for the medicine.  The submission deadline is Monday 30th June 2025. 

The SMC will then review all the evidence submitted by different stakeholders: 

• Information from the pharmaceutical company. 
• Information from clinicians.   
• Real-world experiences collected by Patient Groups  
• The output from a Patient and Clinician Engagement (PACE) meeting. 

This combined evidence will inform a decision on routine use of the medicine in NHS Scotland.  

 The SMC said to SMA UK: 

 “It is very important that we capture the impact of this condition and the potential benefit this medicine has to both patients and carers.” 

“..the wealth of knowledge and experience your organisation could provide would help our SMC Committee members to make a fully informed decision on whether they can make this medicine available on the NHS to patients in Scotland. “  

 How you can help 

It is crucial that we gather the thoughts and experiences from people living with SMA in Scotland.  

If you live with SMA in Scotland, you can:  

• Complete our treatment experience survey, allowing us to share insightful real-world experiences with the SMC committee.  

Survey Link: 

https://forms.office.com/e/sYccAsaqQm 

• Volunteer as a patient expert. If you or someone you care for: 

1. Lives with SMA type 2 or 3 in Scotland 
2. Has had experience of accessing Spinraza in the UK 

Then you can volunteer to participate on the as a patient expert at the Patient and Clinician Engagement (PACE) meeting.  

For more information on the patient expert role please contact portia.thorman@smauk.org.uk 

July: SMC announces nusinersen can now be prescribed for all those who have SMA Types 2 or 3 under the ultra-orphan pathway for a period of up to three years while further evidence on its effectiveness is generated.

6th February: SMA UK makes submission to the Scottish Medicines Consortium which includes the summary of results of our survey of community views.

13th March: Kathy Huffman (who, with her husband, tried to gain access to the Type 1 Expanded Access to Nusinersen Programme for her daughter who sadly died a year ago) joined Sheonad MacFarlane, whose daughter has SMA Type 2, to make the patient presentation to the Scottish Medicines Consortium (SMC) Patient and Clinical Engagement (PACE) meeting. Their testimonies were very powerful and moving and we are very grateful to them for taking this on. SMA Support UK’s Support Services Manager, the SMA Trust’s CEO, and Muscular Dystrophy UK’s Campaigns and Engagement Manager, were also there to support them and answer any questions. We worked together to make a strong case for access.

7th May: the SMC committee’s announcement stated:

"the committee heard compelling evidence on the benefits of nusinersen, supported by the views of experts and patient groups and was able to apply a very high degree of flexibility in accepting this medicine for type 1 SMA. Unfortunately, the evidence presented suggested that nusinersen was substantially less cost effective when used in types 2 and 3 and therefore could not be accepted by the committee. These are incredibly difficult decisions. The medicine is extremely expensive and there is a need to consider all those who need treatment by NHS Scotland. We would welcome a resubmission from the company which addresses the issues raised.”

Dr Sheonad MacFarlane’s daughter, Eilidh, nine, has SMA Type 2. Sheonad, from Glasgow, also chairs Muscular Dystrophy UK’s Scottish Council. She said:

“Naturally, the Scottish Medicines Consortium’s original decision for restricted use was a huge disappointment. But we vowed to continue fighting, and the decision to make it available for people with all types of SMA now feels like we have been given a second chance. Having access to Spinraza means so much to us and to other families, and we are overjoyed that our dreams have finally been realised.

“As a parent, it’s heart-breaking to see your child gradually lose their physical abilities. Nothing can prepare you for the devastating emotional impact this has on you, particularly when there is a treatment out there that is agonisingly out of reach. But now we can look to the future.”

Amy Cameron’s three-year-old son, Zac, has SMA Type 1 and has access to Spinraza on the NHS in Scotland. Amy, from Alloa, Clackmannanshire, said:

“Our whole life was thrown into a whirlwind the day we heard those three words – spinal muscular atrophy. But Spinraza has bought us valuable time with our little boy and given us hope for the future. Zac has achieved things we never thought possible and continues to amaze us every day.

“I’m so pleased the Scottish Medicines Consortium has done the right thing and approved Spinraza for people with other types of SMA. It’s made such a difference to our lives, and other families deserve that same chance.”

8th May: SMA UK sends letter to the Cabinet Secretary for Health and Sport to ask that Dr Brian Montgomery’s 2016 Review of Access to New Medicines Report recommendation for the development of an alternative assessment pathway for ultra-orphan medicines be implemented urgently so that nusinersen could be reassessed for Types 2 and 3 under the new process as soon as possible.

16th May: Patient groups send joint letter to Biogen to ask them to review their pricing, work further on gathering evidence and re-submit as soon as possible. Biogen replies.

8th October: Ultra-orphan pathway now in place